The National Institute for Health and Care Excellence (NICE) has recommended Pedmarqsi (anhydrous sodium thiosulfate) for the prevention of cisplatin-induced ototoxicity (CIO) in paediatric patients aged 1 month to 17 years with localised, non-metastatic, solid tumours.
Cisplatin, a potent chemotherapy agent, is widely used in treating childhood cancers. However, it carries a significant risk of causing irreversible, bilateral hearing loss, known as ototoxicity, in approximately 60% of children. This hearing loss can begin with the first dose of cisplatin and worsen with subsequent doses, profoundly impacting a child's speech, language acquisition, psycho-social and cognitive development, and overall quality of life.
Mark Ashton of Norgine Pharmaceuticals commented:
“We are delighted that the National Institute for Health and Care Excellence (NICE) has recommended Pedmarqsi, highlighting the value it brings to this important unmet medical need. It also recognises the positive impact that this treatment could have not only on the quality of life, but importantly, on the future of these children. At Norgine, we are excited to build our presence in paediatric oncology, as we aim to bring transformative and life-changing medicines to children in the UK.”
Pedmarqsi is the first and only treatment specifically designed to prevent ototoxicity in paediatric patients with localised solid tumours. NICE's recommendation marks a significant milestone, offering hope to young patients and their families. The decision is supported by data from two open-label, randomized Phase III trials, SIOPEL 6 and the Clinical Oncology Group (COG) Protocol ACCL0431. These studies demonstrated that anhydrous sodium thiosulfate (STS) reduced the incidence of cisplatin-induced hearing loss by approximately 50% compared to cisplatin alone.
With 283 new cases of ototoxic hearing loss diagnosed in individuals under 18 in England from 2022 to 2023, the adoption of Pedmarqsi could significantly reduce the long-term developmental and quality-of-life impacts on affected children.
NICE's endorsement of Pedmarqsi represents a pivotal advancement in paediatric cancer care, providing a much-needed solution to mitigate the debilitating side effects of cisplatin.
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